After Ultomiris stumble, Alexion delivers a rare disease win for AZ

A drug for Wilson disease developed by AstraZeneca’s Alexion rare disease unit has cleared a phase 3 trial, setting up regulatory filings in the coming months.

The FoCus study of ALXN1840 showed that once-daily oral drug met its primary objective in improving a key biomarker in Wilson disease, a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised allowing it to build to toxic levels.